Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A (FANCOLEN-1)
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|ClinicalTrials.gov Identifier: NCT03157804|
Recruitment Status : Unknown
Verified November 2020 by Julian Sevilla, Hospital Infantil Universitario Niño Jesús, Madrid, Spain.
Recruitment status was: Active, not recruiting
First Posted : May 17, 2017
Last Update Posted : November 25, 2020
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This is an open, Phase I / II clinical trial to evaluate the safety and efficacy of a hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector carrying the FANCA gene for patients with Fanconi Anemia of Subtype A .
CD34 + cells derived from bone marrow and / or mobilized peripheral blood (fresh and / or cryopreserved) from patients with Fanconi subtype A (FA-A), will be transduced ex vivo with a lentiviral vector carrying the gene FANCA (orphan drug) . After transduction the cells will be inoculated in patients in order to restore their hematopoiesis with genetically corrected stem cells.
|Condition or disease||Intervention/treatment||Phase|
|Fanconi Anemia||Procedure: IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs) Biological: Genetically Engineered Hematopoietic Stem/Progenitor Cells Other: Laboratory Biomarker Analysis Biological: Filgrastim Drug: Plerixafor Procedure: Bone Marrow Aspiration||Phase 1 Phase 2|
The main objective of this open-label Phase I / II clinical trial is to evaluate the safety and therapeutic efficacy of a hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector carrying the FANCA gene for patients with Fanconi's Anemia Subtype A.
The drug to be administered to the patients consists of the cellular product resulting from the transduction of autologous CD34 + cells with the therapeutic lentiviral vector PGK-FANCA.Wpre *.
The dose of cells to infuse in the patients will be that obtained from the transduction process of between 3x10^5 and 4x10^6 CD34 + cells / kg of patient body weight.
The cells will be infused intravenously in a single dose, after complete the transduction process.
Follow-up period: 3 years after infusion of transduced cells. However, patients will be monitored outside the clinical trial over a 10-year period.
Follow-up of the grafted transduced cells will be performed on peripheral blood and bone marrow samples.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||9 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Clinical Trial Phase I / II to Evaluate the Safety and Efficacy of the Infusion of Autologous CD34 + Cells Transduced With a Lentiviral Vector Carrying the Gene FANCA in Patients With FA Subtype A (FANCOLEN-1)|
|Actual Study Start Date :||January 7, 2016|
|Actual Primary Completion Date :||April 23, 2019|
|Estimated Study Completion Date :||April 2022|
Experimental: Autologous CD34+ cells transducted with PGK-FANCA-Wpre *
CD34 + cells from patients with Fanconi subtype A (FA-A) transduced ex vivo with lentiviral vector carrying the gene FANCA, PGK-FANCA-Wpre*The product to be infused consist of a suspension of transduced CD34^+ cells.
Procedure: IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs)
Biological: Genetically Engineered Hematopoietic Stem/Progenitor Cells
Undergo infusion of genetically modified hematopoietic progenitor cell therapy
Other Name: Genetically Engineered HSPCs
Other: Laboratory Biomarker Analysis
Given subcutaneously (SC)
Other Name: Filgrastim XM02, Filgrastim-sndz, G-CSF (Colony Stimulating Factor), Neupogen, r-metHug-CSF, Recombinant Methionyl Human Granulocyte CSF, rG-CSF, Tbo-filgrastim, Zarxio
Other Name: AMD 3100, JM-3100, Mozobil, SDZ SID 791
Procedure: Bone Marrow Aspiration
- Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: Up to 3 years after infusion of transduced cells ]All adverse events will be registered for 3 years from infusion of transduced cells
- Proportion of patients with at least 0.1 copy of the therapeutic vector per nucleated bone marrow or peripheral blood cells three years after infusion. [ Time Frame: 3 years after infusion of transduced cells ]Detection of at least 0.1 copy of the therapeutic vector per nucleated bone marrow cell or peripheral blood cells three years after infusion.
- Proportion of patients with clinical hematological response after the infusion of autologous CD34 + cells transduced with the therapeutic lentiviral vector [ Time Frame: 3 years after infusion of transduced cells ]Proportion of patients with clinical hematological response (improvement of cell blood counts at least in one hematological lineage).
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|Ages Eligible for Study:||1 Year to 21 Years (Child, Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Patients diagnosed with Fanconi Anemia complementation group A (FA-A)
- Minimum age 1 year
- Maximum age 21 years
- Lansky Index> 60%.
- Informed consent in accordance with current legal regulations.
- Number of cells to be transduced: At least 3x10^5 purified CD34+ / kg body weight.
- Negative result in the urine pregnancy test at the baseline visit for women of childbearing age, who should be committed to using an effective contraceptive method during the period of study participation.
- Patients with an human leukocyte antigen (HLA) identical family donor.
- Evidence of myelodysplastic syndrome or leukemia, or cytogenetic abnormalities predicting the same in bone marrow aspirates. In this case, the studies carried out two months in advance of the patient's entry into the clinical trial will be considered valid.
- Evidence that the patient to be infused has signs of somatic mosaicism, with hematologic improvement.
- Any illness or concomitant process that in the opinion of the investigator incapacitates the subject for their participation in the study.
- Pre-existing sensory or motor impairment> = grade 2 according to the National Cancer Institute (NCl) criteria.
- Pregnant or lactating women.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03157804
|Hospital Vall d'Hebron|
|Barcelona, Spain, 08035|
|Hospital Infantil del Niño Jesus|
|Madrid, Spain, 28009|
|Study Director:||Juan A Bueren||CIEMAT/CIBERER/IIS.FJD|
|Responsible Party:||Julian Sevilla, M.D.PhD Specialist in Hematology Hemotherapy, Responsible for the Transfusion Service and Unit for the Obtention and Processing of Hematopoietic Progenitors and other cellular therapies at the Hospital Infantil Universitario Niño Jesús de Madrid., Hospital Infantil Universitario Niño Jesús, Madrid, Spain|
|Other Study ID Numbers:||
|First Posted:||May 17, 2017 Key Record Dates|
|Last Update Posted:||November 25, 2020|
|Last Verified:||November 2020|
Anemia, Hypoplastic, Congenital
Congenital Bone Marrow Failure Syndromes
Bone Marrow Failure Disorders
Bone Marrow Diseases
Genetic Diseases, Inborn
DNA Repair-Deficiency Disorders
Renal Tubular Transport, Inborn Errors
Female Urogenital Diseases
Female Urogenital Diseases and Pregnancy Complications
Male Urogenital Diseases
Physiological Effects of Drugs